How a funny-looking creature could unlock the secrets of limb regeneration

The axolotl, also known as the Mexican salamander

In the world of funny-looking creatures, the Axolotl would have to rank in the top ten alongside such notables as the naked mole rat and the blob fish (the official mascot for the Ugly Animal Preservation Society). But the Axolotl does have one attribute that makes it attractive to more than just another Axolotl. That’s because this Mexican salamander has the ability to regenerate entire limbs.

Now, even as you read this, many stem cell researchers are hard at work trying to figure out ways to regenerate damaged or diseased tissues and organs in humans. That’s why the Axolotl is so intriguing. If we can understand how they are able to repair their own damaged limbs, maybe we can use that knowledge to help people repair or even replace a lost finger, hand or arm.

It’s a fascinating idea and one that is explored in this video from STAT, an online publication produced by the Boston Globe, that explores science and health.

It’s only four minutes long and is definitely worth watching. It shows that there is beauty in even the strangest creatures, if only you know what to look for.

How mice and zebrafish are unlocking clues to repairing damaged hearts

Bee-Gees

The Bee Gees, pioneers in trying to find ways to mend a broken heart. Photograph: Michael Ochs Archives

This may be the first time that the Australian pop group the Bee Gees have ever been featured in a blog about stem cell research, but in this case I think it’s appropriate. One of the Bee Gees biggest hits was “How can you mend a broken heart” and while it was a fine song, Barry and Robin Gibb (who wrote the song) never really came up with a viable answer.

Happily some researchers at the University of Southern California may succeed where Barry and Robin failed. In a study, published in the journal Nature Genetics, the USC team identify a gene that may help regenerate damaged heart tissue after a heart attack.

When babies are born they have a lot of a heart muscle cell called a mononuclear diploid cardiomyocyte or MNDCM for short. This cell type has powerful regenerative properties and so is able to rebuild heart muscle. However, as we get older we have less and less MNDCMs. By the time most of us are at an age where we are most likely to have a heart attack we are also most likely to have very few of these cells, and so have a limited ability to repair the damage.

Michaela Patterson, and her colleagues at USC, set out to find ways to change that. They found that in some adult mice less than 2 percent of their heart cells were MNDCMs, while other mice had a much higher percentage, around 10 percent. Not surprisingly the mice with the higher percentage of MNDCMs were better able to regenerate heart muscle after a heart attack or other injury.

So the USC team – with a little help from CIRM funding – dug a little deeper and did a genome-wide association study of these mice, that’s where they look at all the genetic variants in different individuals to see if they can spot common traits. They found one gene, Tnni3k, that seems to play a key role in generating MNDCMs.

Turning Tnni3K off in mice resulted in higher numbers of MNDCMs, increasing their ability to regenerate heart muscle. But when they activated Tnni3k in zebrafish it reduced the number of MNDCMs and impaired the fish’s ability to repair heart damage.

While it’s a long way from identifying something interesting in mice and zebrafish to seeing if it can be used to help people, Henry Sucov, the senior author on the study, says these findings represent an important first step in that direction:

“The activity of this gene, Tnni3k, can be modulated by small molecules, which could be developed into prescription drugs in the future. These small molecules could change the composition of the heart over time to contain more of these regenerative cells. This could improve the potential for regeneration in adult hearts, as a preventative strategy for those who may be at risk for heart failure.”

 

 

 

Fujifilm is Expanding Its Focus to Regenerative Medicine

Fujifilm began as a photography company, but today is a well-known multinational imaging and information technology corporation. More recently, it’s expanded its focus (pun intended) on developing innovative technologies in the healthcare and regenerative medicine space.

The news that Fujifilm was expanding into regenerative medicine was surprising to some given the company’s expertise in areas unrelated to stem cell research, but with the acquisition of Cellular Dynamics International, a company from Madison, Wisconsin that specializes in large-scale manufacturing of human cells, and the revamping of Fujifilm’s Japan Tissue Engineering subsidiary, which is developing regenerative treatments for damaged skin and cartilage, Fujifilm has solidified its position as a competitive company that’s accelerating the pace of regenerative medicine to develop treatments for patients with unmet medical needs.

Mr. Ban

Mr. Toshikazu Ban

So what progress has Fujifilm made in regenerative medicine and what advancements are they making towards the clinic? You’ll find the answers to these burning questions in my interview with Mr. Toshikazu Ban, Corporate Vice President, General Manager of Regenerative Medicine Business Division at Fujifilm Corporation. Enjoy!

Q: Why did Fujifilm decide to enter the regenerative medicine space?

TB: At first glance, Fujifilm may seem an unlikely candidate to become a leader in regenerative medicine, yet its engagement in the healthcare industry goes back many decades. Founded in 1934, Fujifilm started offering X-ray film just two years later. By 1983, Fujifilm became the first in the world to offer a digital X-ray diagnostic imaging system.

Today, Fujifilm has been able to expand the use of its core fundamental technologies in cosmetics and supplements and pharmaceuticals. Combined, these have allowed Fujifilm to transform into a major healthcare company committed to prevention, diagnosis and treatment.

Unfortunately, there are still many diseases for which there are no effective treatments, and millions wait in hope of their discovery. Regenerative medicine treatment has the potential to cure diseases that cannot be cured by drugs. Fujifilm feels a sense of responsibility to apply its technology in a way that helps make promising treatments a reality.

Q: What advantages do you think Fujifilm has over other healthcare companies in regenerative medicine?

TB: Fujifilm’s advanced engineering technology provides tremendous possibilities in the regenerative medicine space.

The chief component in photographic film is gelatin, which is derived from collagen. Fujifilm has developed a human-type recombinant peptide which can be scaffolds for growing cells and restoring tissue.  The human-type recombinant peptide is non-animal based, has high cellular adhesiveness, is flexible, safe, biocompatible, biodegradable and bioabsorbable. Cells survive better when they are combined with our recombinant peptide because it holds the cells better and allows space in between so that oxygen and other critical growth factors can reach the cells.

Fujifilm also has two subsidiaries that provide synergies and efficiencies to be more competitive in the regenerative medicine field, Cellular Dynamics International, Inc., (FCDI), and Japan Tissue Engineering Co., Ltd. (J-TEC).

In 2015, FCDI announced the launch of a stem cell bank with funding from CIRM to create induced pluripotent stem (iPS) cell lines for each of 3,000 healthy and diseased volunteer donors across 11 common diseases and disorders to be made available through the CIRM human pluripotent stem cell (hPSC) Repository.

The lines available from the CIRM stem cell bank directly complement FCDI’s ability to provide differentiated cells corresponding to each of the iPSC lines, which will allow researchers to model the diseases represented, better understand disease progression, perform more targeted drug discovery, and ultimately lead to better treatments.

A lot of pharmaceutical companies use these cells to test for the screening and toxicity of new drug candidates. If iPS cells can improve the productivity including efficacy and safety, the technology can greatly reduce time and cost as well as the drop-out rate in clinical development.

In 2014, J-TEC became a consolidated Fujifilm Group subsidiary. J-TEC launched the first two regenerative medicine products to receive approval from the Japanese government (one product is used to treat severe burns, while the other is used to replace damaged cartilage in knees).

J-TEC Lab (Image courtesy of Fujifilm)

J-TEC Lab (Image courtesy of Fujifilm)

Q: Can you describe some of the stem cell therapies you’re developing for the clinic for major diseases?

TB: FCDI plans to start iPS cell therapy clinical studies in the U.S. for age related macular degeneration in the year 2017, and clinical studies for retinitis pigmentosa, Parkinson’s and heart failure around 2019.

In March 2015, Fujifilm announced it had developed diabetes therapies in animal tests. CellSaic is a three-dimensional mosaic structure that combines cells with a recombinant peptide (RCP) scaffold made from micro-sized petaloid pieces of the protein. In a study involving type 1 diabetic mice, we created a CellSaic of human mesenchymal stem cells and cells from pancreatic islets and transplanted them in the mice. The purpose of the study was to verify whether using the recombinant peptide as a scaffold would increase the survival rate of the transplanted cells compared with just transplanting the cells alone. We also wanted to demonstrate a reduction in blood glucose levels of the diabetic mice since the recombinant peptide was able to sustain the viability of the pancreatic islet cells.

The study showed that seven days after the transplantation, CellSaic had a significantly more prominent introduction of blood vessels, which provide passageways for nutrients, oxygen and waste product to get to, and away from, the cells.  In addition, 28 days after transplantation, the test group of diabetic mice with the recombinant peptide-based CellSaic scaffold saw blood glucose levels lowered to the level equivalent to that of the healthy mice. In contrast, the diabetic mice who received pancreatic islets alone showed no change in blood glucose levels. 

Q: When you move into clinical trials, do you anticipate US trial sites in parallel with those in Japan?

TB: FCDI plans to start clinical trials of iPS cell treatments in the US. J-TEC conducts clinical trials for autologous cultured corneal epithelium and plans to start clinical trials for allogeneic cultured dermis in Japan. Currently we plan to conduct these clinical trials where these companies are located. We may expand the clinical trials of the products to other countries in the future.

Q: Can you speak to Japan’s regulatory system for stem cell therapies and how this could give Fujifilm a leg up on developing stem cell treatments more rapidly?

TB: The go-to market conditions for regenerative medicine in Japan have become more favorable since the November 2014 implementation of the Pharmaceutical and Medical Device Law, which has significantly cut the time it takes to gain marketing approval in Japan and created more interest in this sector.

Within regenerative medicine, academic institutions have shown remarkable progress. The mission of the industry is to apply findings from academia to patients and deliver high-quality treatments at a reasonable cost.

Note: Technologies that pertain to Japan Tissue Engineering Co., Ltd. (J-TEC) are not approved for use in the US.

You can learn more about Fujifilm’s latest efforts to “make regenerative medicine a reality” by visiting its Innovation website.

Getting On Tract: Stem Cells Regenerate Injured Spinal Cord in Rats

a6353-spinalcordThe spinal cord acts as a highway that transports electrical signals from your brain to the rest of your body through long bundles of nerve fibers. It allows your brain to communicate with the rest of your body to coordinate movement and reflexes and to receive sensory information. When the spinal cord is damaged, the nerve fibers, which are also called axons, are crushed or severed. This important communication highway is disrupted, leaving patients partially or fully paralyzed and severely reducing their quality of life.

Stem cell treatments for spinal cord injury

Scientists are pursuing multiple strategies using stem cells to treat spinal cord injury. Some involve transplanting cells derived from pluripotent stem cells or from stem cells isolated from human tissue. Both types of stem cells can be manipulated to develop into new nerve cells that can replace those that have died or into new support cells that coax damaged nerve cells to regrow their axons. Others are transplanting stem cells at the site of injury to prevent further damage by reducing tissue scarring and inflammation or by releasing protective factors that keep the remaining nerve and support cells healthy.

Repairing a damaged spinal cord is no easy task. Stem cell treatments tested in animal models have only shown partial recovery of motor function, and clinical trials using stem cells to treat spinal cord injury in humans are still in their early stages (read more about clinical trials here).

However, a group from UC San Diego is on “tract” (pardon the pun) to develop a novel treatment that might one day regenerate damaged spinal cords in humans. They published their exciting study in Nature Medicine yesterday suggesting that stem cells can regenerate injured spinal cords – at least in rats.

Getting on tract to regenerate injured spinal cords

The team grafted neural stem cells derived from rat embryonic stem cells into the injured spinal cords of rats. These stem cells developed into functional nerve cells that replaced damaged axons in the corticospinal tract, which is bundle of nerve fibers in the spinal cord that originates in the cerebral cortex of the brain and controls basic motor function. Injured rats that received stem cell grafts showed improvements in their ability to move their forelimbs.

Transplanted neural stem cells (green) shown in the corticospinal tract (red) develop into new neurons (purple). (Nature Medicine)

Transplanted neural stem cells (green) shown in the corticospinal tract (red) develop into new neurons (purple). (Nature Medicine)

The authors also grafted neural stem cells derived from human embryonic stem cells into injured spinal cords of rats and observed evidence of spinal cord regeneration and newly generated corticospinal axons.

The study’s senior author, Dr. Mark Tuszynski, explained in a UC San Diego Health news release that their study is the first to regenerate the corticospinal tract in rats using neural stem cells. While this work is in its early stages, Dr. Tuszynski believes that his group’s work has the potential to be translated into a treatment for human spinal cord injury:

Mark Tuszynski, UCSD

Mark Tuszynski, UCSD

“We humans use corticospinal axons for voluntary movement. In the absence of regeneration of this system in previous studies, I was doubtful that most therapies taken to humans would improve function. Now that we can regenerate the most important motor system for humans, I think that the potential for translation is more promising.”

 

However, when translating any stem cell therapy from animals into humans, safety and efficacy are a top priority. Dr. Tuszynski acknowledged these hurdles and shared his plan for future studies:

“There is more work to do prior to moving to humans. We must establish long-term safety and long-term functional benefit in animals. We must devise methods for transferring this technology to humans in larger animal models. And we must identify the best type of human neural stem cell to bring to the clinic.”

As a side note, CIRM has funded earlier translational research by Dr. Tuszynski. You can read more about his CIRM-funded research project to develop novel stem cell treatments for spinal cord injury here.


Related Links:

How you derive embryonic stem cells matters

A scientist named James Thompson was the first to successfully culture human embryonic stem cells in 1998. He didn’t know it then, but his technique isolated a specific type of embryonic stem cell (ESC) that had a “primed pluripotent state”.

There are actually two phases of pluripotency: naïve and primed. Naïve ESCs occur a step earlier in embryonic development (during the beginning of the blastocyst stage), and the naïve state can be thought of as the ground state of pluripotency. Primed ESCs on the other hand are more mature and while they can still become every cell type in the body, they are somewhat less flexible compared to naïve ESCs. If you want to learn more about naïve and primed ESCs, you can refer to this scientific review.

Scientists have developed methods to derive both naïve and primed human ESCs in culture and are attempting to use these cells for biomedical applications. However, a recent CIRM-funded study published in Cell Stem Cell, calls into question the quality of ESCs produced using these culturing methods and could change how lab-derived stem cells are used for stem cell transplant therapies and regenerative medicine.

Primed human embryonic stem cells (purple) identified by a green stem cell surface marker. (Image courtesy of UCLA)

Primed human embryonic stem cells (purple) identified by a green stem cell surface marker. (Image courtesy of UCLA)

Culturing methods erase stem cell memory

UCLA scientists discovered that some of the culturing methods used to propagate naïve ESCs actually erase important biochemical signatures that are essential for maintaining ESCs in a naïve state and for passing down genetic information from the embryo to the developing fetus.

When they studied naïve ESCs in culture, they focused on a naturally occurring process called DNA methylation. It controls which genes are active and which are silenced by adding chemical tags to certain stretches of DNA called promoters, which are responsible for turning genes on or off. This process is critical for normal development and keeping cells functional and healthy in adults.

UCLA scientists compared the DNA methylation state of the mature human blastocyst – the early-stage embryo and where naïve ESCs come from – to the methylation state of naïve ESCs generated in culture. They found that the methylation patterns in the blastocyst six days after fertilization were the same as the patterns found in the egg that it developed from. This discovery is contrary to previous beliefs that the DNA methylation patterns in eggs are lost a few hours after fertilization.

Amander Clark, the study’s lead author and UCLA professor explained in a UCLA news release:

Amander+Clark+headshot_68295d00-2717-4d5c-99f3-f791e6b6ebcf-prv

Amandar Clark, UCLA

“We know that the six days after fertilization is a very critical time in human development, with many changes happening within that period. It’s not clear yet why the blastocyst retains methylation during this time period or what purpose it serves, but this finding opens up new areas of investigation into how methylation patterns built in the egg affect embryo quality and the birth of healthy children.”

The group also discovered cultured naïve ESCs lack these important DNA methylation patterns seen in early-stage blastocysts. Current methods to derive naïve ESCs wipe their memory leaving them in an unstable state. This is an issue for researchers because some prefer the use of naïve ESCs over primed ESCs for their studies because naïve ESCs have more potential for experimentation.

“In the past three years, naïve stem cells have been touted as potentially superior to primed cells,” Clark said. “But our data show that the naïve method for creating stem cells results in cells that have problems, including the loss of methylation from important places in DNA. Therefore, until we have a way to create more stable naïve embryonic stem cells, the embryonic stem cells created for the purposes of regenerative medicine should be in a primed state in order to create the highest-quality cells for differentiation.”

How you derive embryonic stem cells matters

Now that this culturing problem has been identified, the UCLA group plans to develop new and improved methods for generating naïve ESCs in culture such that they retain their DNA methylation patterns and are more stable.

The hope from this research is that scientists will be able to produce stem cells that more closely resemble their counterparts in the developing human embryo and will be better suited for stem cell therapies and regenerative medicine applications.


Related Links:

What Went Down at ARM’s Regenerative Medicine State of the Industry

Every January, downtown San Francisco is taken over by a flock of investors, bankers, biotech companies, and scientists attending the annual JP Morgan Healthcare Conference. This meeting looks at the healthcare advancements over the past year and predicts the disease areas and technologies that will see the most progress and success in 2016.

According to some of the experts at the event, regenerative medicine and stem cell research are experiencing impressive, accelerated advancements, which has peaked the interest of investors, biotech, and pharmaceutical companies.

Because these are such fast paced fields, the Alliance for Regenerative Medicine (ARM) hosts the Annual Regenerative Medicine and Advanced Therapies State of the Industry Briefing during JP Morgan to discuss the recent progress and outlook for the industry in the coming year.

Screen Shot 2016-01-11 at 4.03.30 PM

What happened in 2015 and what’s next?

ARM’s  6th Annual Briefing was open to the public and drew over 300 people on Monday morning. The meeting opened with an industry update from Edward Lanphier, ARM Chairman and President/CEO of Sangamo BioSciences.  Then two panels featuring top leaders from biotech and pharmaceutical companies discussed the 2016 clinical data forecast and the promise of regenerative medicine and advanced therapies in oncology (cancer).

With an upbeat attitude, Lanphier gave an overview of clinical development progress in 2015, with 20 approved products worldwide and over 600 clinical trials both from academia and industry. More than 40% of these ongoing clinical trials are in cancer while approximately 12% are in heart disease/injury. These trials are not limited to Phase 1 either. In 2015, there were 376 in Phase 2 (compared to 200 in 2014) and 64 in Phase 3 (compared to 39 in 2014).

Edward Lanphier

Edward Lanphier

Two other areas Lanphier emphasized were CAR-T and other cell-based immunotherapies and gene therapy programs for rare diseases. He ended with 2015 statistics on clinical milestones in various disease and therapy programs, key company IPOs, the financial landscape, and predictions of major anticipated data from clinical trials in 2016.

It was a lot to take in, but this was definitely a good thing and a sign that the areas of regenerative medicine and advanced therapies are thriving. If you want more details, you can check out ARM’s State of the Industry presentation.

Major Theme: Data is King

The major theme that cropped up during the industry update and panel discussions was the importance of producing meaningful clinical data to get positive outcomes in regenerative medicine.

This was succinctly put by panelist Sven Kili, head of Gene Therapy Development at GlaxoSmithKline:

“I would say “Data is King”. A great idea is fantastic, passion is wonderful, and most companies will buy into a strong management team, but that only gets you so far. After that you need to have data, and you need to have a good plan for going forward.”

Kill added that there’s the need to work with the FDA to change the regulatory process, saying the FDA is, understandably, cautious about working with therapies that can alter a person’s genome permanently. However, he said there needs to be serious discussions with the FDA about how to speed up the process, to make it easier for the most promising projects to get approval.

Edward Lanphier also talked about the industry’s new focus on clinical data and the questions that arise when trying to advance regenerative medicine research into approved treatments and cures for patients:

“How do we communicate the value of curing blindness? How do we think about pricing that? What do we think about [drug] reimbursement?  For rare diseases, we aren’t trying to talk about acute treatments – we are talking about one-time, curative outcomes. And the value and benefit to patients in this is enormous. This is what we are trying to do, and on the cusp of, in terms of generating both approvable data and also the proof of concept data that then allows us to drive that next value inflection point in terms of financings.”

The Future Looks Good

After listening to the briefing, the future of regenerative medicine and advanced therapies certainly looks bright. As Jason Kolbert, head of Healthcare Research at the Maxim Group, said:

“This industry is now rapidly maturing and regenerative medicine and gene therapy have great things in store for the next decade.”

Usman Azam, Global Head of Cell and Gene Therapies at Novartis, had a similar outlook:

“We now are going from proof of concept to commercial availability of a disruptive innovation within seven years. If somebody had said that to me four years ago, I would have said, not possible. But that gives you a sense of how quickly this field is moving.”

Experts Panel

ARM Panel: 2016 Sector Forecast: Upcoming Clinical Data Events

Creative partnerships that promote progress

Lewis and Clark: great partnerships can change the world

Lewis and Clark: great partnerships can change the world

Having a good partner can turn something good into something truly memorable. Where would Laurel be without Hardy, Lewis without Clark, Butch Cassidy without the Sundance Kid. That’s why the stem cell agency has partnerships on a number of different levels as part of our mission of accelerating the development of stem cell cures to patients with unmet medical needs.

Our latest partnership is with RegMedNet which, in its own words, “provides a unique and unparalleled platform for the regenerative medicine community to share insights, discuss the latest research, and help move the field forward.” With a goal like that why would we not want to support them?

Like us RegMedNet believes that regenerative medicine is going to completely change the way we treat disease, even the way we think about disease. They also believe that progress of the kind we all want is only going to come by bringing together all the key players from the researchers and manufacturers, to the government regulators and, of course, the patient advocates. Each has a vital role to play in moving the field forward and RegMedNet reflects that in both the content it posts online and in the contributors, who represent institutions and companies worldwide.

One of the most important elements in any partnership is understanding, and RegMedNet does a great job of trying to raise awareness about the field, the challenges we all face, and the progress being made. Bringing together so many different perspectives in one spot really helps create a much deeper understanding of regenerative medicine as a whole.

In a few short years regenerative medicine has gone from a relatively small field to a global industry. Our hope is that creating partnerships with like-minded groups around the world, is going to help it get even bigger and, even better.

How one strong ARM can create a community

I spent the last two days at the annual Washington meeting of the Alliance for Regenerative Medicine (ARM), the advocacy organization that CIRM became a founding member of in 2009. Having been CIRM’s representative at that first organizing meeting it has been a pleasure to see the organization mature into an effective advocacy group for our field. It has lived up to its goal of creating a community where all the stakeholders in the field, from academic and industry leaders to patient advocates and investors, can come together in a coordinated front.

ARM and CIRM share the goal of accelerating the development of regenerative therapies to patients with unmet medical needs. The organization also dovetails well with our effort to inform the public about the great hope in the field. To quote ARM’s website: “ARM also works to increase public understanding of the field and its potential to transform human healthcare.”

But that transformation can be fostered or impeded by actions in our nation’s capital, both regulatory and legislative, the main thrust of the past two days’ activities.

While the iconic Capitol building is the most recognized footprint of our Congress, it is the House and Senate office buildings that ring three sides of the Capitol where most of the work gets done, like in the Rayburn building, which houses the office of Dianna DeGette, the Colorado congresswoman and champion of regenerative medicine.

While the iconic Capitol building is the most recognized footprint of our Congress, it is the House and Senate office buildings that ring three sides of the Capitol where most of the work gets done, like in the Rayburn building, which houses the office of Dianna DeGette, the Colorado congresswoman and champion of regenerative medicine.

ARM members presented three specific proposals for advancing the field to members of congress and their staffs. These would:

  • Create a center of excellence to develop technical and process standards for regenerative medicine. Not very sexy on the surface, but agreement in advance on what regulators will accept in creating a new product can shave months or years off the development of needed therapies.
  • Create a special pathway within the Food and Drug Administration—much like the one created for orphan diseases—for “Qualified Regenerative Medicine Products (QRMPs). These products would have shown potential to change the course of a disease with currently unmet medical needs and the FDA would be required to meet with their sponsors to discuss expedited review of the product.
  • Advocate for the adoption of a national regenerative medicine strategy that includes federal agency coordination, support for research and regulatory reform to create a clear and predictable pathway that enables quick approval of safe and effective products. To accomplish that ARM has promoted the establishment of a Regenerative Medicine Coordinating Council within the U.S. Department of Health & Human Services.
Jamie Goldfarb with her son Kai and husband Jeff. Photo courtesy Melanoma Research Alliance

Jamie Goldfarb with her son Kai and husband Jeff.
Photo courtesy Melanoma Research Alliance

Jamie Goldfarb, who beat back melanoma with the help of a cell-based immune therapy, made a clear and passionate case for the urgency of making it easier to get these therapies to patients at the ARM member dinner Tuesday night:

“Enhanced awareness for the power of regenerative medicine means a world of difference. It means less suffering, less pain, less fear, less expense, less hardship, less loss. It also means more hope, more determination, more love, more strength for individuals and for society as a whole. Every person in this room and those organizations you represent are improving lives.”

Don Gibbons

Regenerative Medicine Takes the Spotlight at this Year’s Largest Biotechnology Convention

BIO logo

It is time to take regenerative medicine seriously. The world’s most inclusive web site for listing clinical trials, clinicaltrials.gov, now has more than 4,000 stem cell trials posted. And the world’s largest biotechnology convention, BIO International, when it kicks off its 2014 edition in San Diego later this month, will include the first ever all-day forum on regenerative medicine—something that has not got much more than a passing nod at previous gatherings.

The BIO leadership asked CIRM to organize the day, and as the principal planner (though with a Y chromosome), I am glad the nine-month gestation is almost over. With the excitement and fear of a soon-to-be new parent I am looking forward to the event Wednesday June 25 at the San Diego Convention Center. I wish I could report that it is open to the public, but it is not. For those who will be at BIO for the week, I hope you will plan to spend that day with us.

We will be presenting five panels with leaders in the field who should provide valuable insights to those new to regenerative medicine as well as those in the thick of trying to accelerate the drive to therapies. Those panels are:

• Regenerative Medicine: Propelling a Paradigm Shift in Medicine and Healthcare Delivery;

• Stem Cells Delivering Results Today as Models of Disease;

• Stem Cells and Gene Therapy, a Great but Challenging Marriage;

• Commercializing a New Therapeutic Modality—Case Studies;

• How International Collaboration Is Accelerating the Field.

These sessions will highlight some of the leading work in California, but also showcase work from around the U.S. and around the world. The speakers will detail the state of the art, but also provide insight into ways their experiences suggest we can accelerate the path to therapies for patients. Finding opportunities to share knowledge gained has always been a central part of CIRM’s mission.

Don Gibbons